Safety and Efficacy of AMT-130 in Adults with HD
Research type
Research Study
Full title
A Phase Ib/II Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington Disease
IRAS ID
288821
Contact name
William Gray
Contact email
Sponsor organisation
uniQure biopharma B.V.
Eudract number
2020-001461-36
Duration of Study in the UK
6 years, 0 months, 16 days
Research summary
Huntington disease (HD) is an illness affecting the brain. It stops parts of the brain from working properly and gets worse over time. Patients may notice changes in thinking, mood, and movement control. Movement changes may include involuntary movements, trouble with walking, talking, and swallowing, and difficulty with activities of daily living. HD is passed on from a person’s parent, and symptoms usually start in people aged between 30 and 50 years old, although it can start sooner or later than this. After a period of up to around 20 years, HD leads to death. There is currently no cure, although some drugs can be used to reduce some of the symptoms.
This research is investigating the use of AMT-130 in people with HD. AMT-130 has not yet been approved by health authorities for regular use but it has been approved for use in this clinical study. AMT-130 is an investigational gene-modified product that it is hoped will lower the level of the protein causing HD in the brain. Participants in the study will receive a dose of AMT-130 and will then be followed-up for 5 years to check the safety of AMT-130 and assess the impact it has on their HD.
uniQure biopharma B.V. (the Sponsor) is responsible for the development and oversight of the study. Up to 15 participants will take part at 3 medical centres in Europe, including the UK.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
20/LO/1190
Date of REC Opinion
5 Mar 2021
REC opinion
Further Information Favourable Opinion